The purpose of this trial is to assess the effect of fenretinide (4-HPR), a vitamin A derivative, in reducing the incidence of breast cancer in healthy premenopausal women at increased familial/genetic risk for breast cancer

Breast cancer incidence [ Time Frame: every 6 months for 15 years ] [ Designated as safety issue: No ]

The aim of the proposed trial is to assess the efficacy of fenretinide, (4 hydroxyphenilretinamide, 4-HPR), a vitamin A derivative, in reducing the incidence of breast cancer (BC) in healthy young premenopausal women at increased familial/genetic risk for BC (i.e. BRCA1 or BRCA2 mutation carriers or subjects at high risk of being carriers). The primary endpoint is the incidence of invasive BC and ductal intraepithelial neoplasia (DIN), histologically diagnosed.

Secondary Outcome Measures:

Incidence of other non-invasive breast disorders, ovarian cancers and other cancers. [ Time Frame: every 6 months for 15 years ] [ Designated as safety issue: No ]

Incidence of other non-invasive breast disorders, ovarian cancers and other cancers.

Early intermediate biomarkers of efficacy after 12, 36, and 60 months of treatment. We will also evaluate the percent change in circulating biomarkers of the IGF system, androgens, retinol binding protein (RBP-4), insulin, blood glucose and VEGF, after 12, 36 and 60 months of treatment.

20-46 years old women with a known BRCA ½ mutation or with a risk of being a mutation carrier ≥20%.

Performance status =0

Willingness to avoid pregnancy during treatment and 12 months after drug cessation

No clinical and radiological evidence of breast cancer and ovarian disease

Signed informed consent

Exclusion Criteria:

History of breast cancer or any other malignancy with the exclusion of CIN and non-melanoma skin cancer

Child bearing or breast feeding

Genetic test result (BRCA)=true negative

Blood test alterations (grade ≥2 based on the NCI Common Toxicity Criteria)

Previous or concurrent use of SERMs, e.g. tamoxifen (for more than 12 months; if less, a two-months wash-out is required before entering the study)

Severe psychiatric disorders or inability to comply to the protocol procedures

Any other factor that, at the investigator's discretion, contraindicates the use of fenretinide

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Please refer to this study by its ClinicalTrials.gov identifier: NCT01479192